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MMRF Blog
June 29, 2009
Perspectives from the DIA Annual Meeting, Anne Quinn Young, MPH
This year was a historic meeting for the Drug Information Association (DIA) – thanks to the visionary perspective of chairperson Nancy Smith, PhD, former Director, Office of Training and Communications, FDA, for the first time, the patient perspective was presented with representation from the MMRF and from the International Alliance of Patients’ Organizations (IAPO).
Last year, Kathy Giusti delivered the keynote address, which led to my participating in two panels to represent the patient perspective on drug development at this year’s meeting. My remarks focused on how the MMRF educates the myeloma community to optimize current therapies as well as ensures that the community has safe and effective therapies in the future. With me on both panels was Ken Kaitin, PhD, Director of the Tufts Center for the Study of Drug Development, who described the MMRF as “changing the landscape in drug development and patient care”. Kathy had the incredible foresight and insight to recognize that the MMRF’s goal mirrored that of industry – to bring new (and of course effective) treatments to patients as quickly as possible – but that was more than 11 years ago! In today’s world, collaboration and transparent communications among all key “pillars” (ie, patients/patient groups, academia, industry, and regulatory authorities) are critical to ensure safe and effective treatments are developed and made available to patients as quickly as possible, and we are more than happy to continue to play the role of congregator and broker to maximize these partnerships.
June 23, 2009
Check Out the Recent Press Release from the MMRC, Anne Quinn Young, MPH, Program Director
Check out the recent press release from the MMRC. We are thrilled to report that we have been able to shave 100 days off of the time to activate a clinical trial in the MMRC as opposed to a published benchmark. This means potential cost savings for our biopharma partners as well as earlier access to promising treatments for patients. By collaborating closely with our MMRC Member Institutions and initiating business solutions such as standardized contracts and processes, as well as site management via the MMRC Multiple Myeloma Program Coordinators (MMPC), we have additional proof that the model works. Stay tuned for the data that show we also complete trials faster!
Read the MMRC Press Release Now: http://www.themmrf.org/about-the-mmrf/powerful-news/press-releases/multiple-myeloma-research-consortium-mmrc-activates-clinical-trials-30-40-percent-faster-than-industry-standard.html
June 3, 2009
Challenges and Opportunities for Personalized Medicine, Susan Kelley, MD, Chief Medical Officer
The data presented at the Annual ASCO meeting on new investigational drugs are of interest to all oncology researchers, and the opportunity to gather with large groups of oncology researchers to review and discuss these results is a critical part of the information exchange process in this field. There were approximately 175 abstracts presented from studies in multiple myeloma, including results from clinical studies ongoing within the MMRC clinical trials network. In addition, there were very informative presentations and discussions on early clinical trials of new drugs against pathways and targets that are relevant to myeloma, even if the study under discussion did not include patients with multiple myeloma. It is these early data that help the researchers in myeloma decide whether to investigate some of these emerging drugs in trials specific for multiple myeloma.
The theme of this year’s congress was “Personalized Medicine “. The entire field of oncology clinical research is at an important crossroads. We now understand enough of the biology of cancers, and have new drugs emerging that allow us to begin to tailor specific drug therapy against the molecular pathways to which a particular tumor, or indeed, a particular patient’s tumor, is especially “addicted” for growth. This will not be a short term effort. The tools for genomic and proteomic analysis need to be applied in a more widespread manner, in carefully constructed clinical trials, with the goal of matching patients with particular drugs or drug combinations. The field of myeloma research is well equipped to pursue this goal of personalized medicine. The scientific and clinical researchers who are our partners in the quest to develop new drugs to improve patient outcomes, and ultimately attain cures, in multiple myeloma, have already been collecting bone marrow samples for important analyses. The next step is to organize a large effort in partnership with our research partners and stakeholders, to design these clinical studies where patient treatment is assigned according to biologic markers and risk factors for a given patient. We cannot stress enough how important it will be for patients to participate in clinical trials as we attempt to move this field forward.
In addition to the efforts around personalized medicine, there were clinical trial results and discussions about important therapeutic issues in myeloma presented at ASCO. For example, with the improved outcomes reported as a result of treatment with the newer drugs that have become available for myeloma in the past 4 years, there are now questions raised about the optimal timing of autologous stem cell transplant—which patients, when and after how much initial therapy? All patients should continue to engage in these important discussions with their physicians.
June 1, 2009
Update from the ASCO Annual Meeting: Cure vs. Control, Anne Quinn Young, MPH, Program Director
On Friday, the MMRF hosted its fifth CME symposium for physicians at the Annual ASCO Meeting. This year, the esteemed faculty included Chair of the Program, Ken Anderson, Dana-Farber Cancer Institute; Sergio Giralt, MD Anderson Cancer Center; Vincent Rajkumar, Mayo Clinic – Rochester; and Paul Richardson, Dana-Farber Cancer Institute. The program was entitled “Treatment of Multiple Myeloma: Cure vs. Control”, based on a thought-provoking paper from Dr. Rajkumar published last year in the Mayo Clinic Proceedings (http://www.mayoclinicproceedings.com/content/83/10/1142.long).
The presentations each addressed the question of whether, at each point in the disease course, the goal of therapy should focus on cure or control. There is consensus on the long-term goal being cure – that much is clear – but at each point, particularly as the disease progresses, there is far from unanimity on how aggressively clinicians should go for the cure – or at least a complete response – at the risk of increased toxicities and impaired quality of life. There does seem to be greater consensus with respect to high-risk patients, as they are more likely to relapse and data indicates that inducing a sustained complete response is critical in these patients. Similarly, there are emerging data to indicate that patients who do not necessarily achieve a CR can benefit from a sustained partial or even minimal response, as shown from a small number of patients who remained stable for a number of years on Revlimid as a single agent – without dex - despite never achieving a CR on therapy. Somewhat counter intuitively, it appears to be more beneficial to achieve a lower but durable response than a transient CR that is associated with rapid relapse (http://www.nature.com/nature/journal/v459/n7246/full/459508a.html).
The question of cure vs. control again emerged subtly in the Sunday morning oral presentations, and it is clear that there is not yet substantial data to help us understand which regimens can maximally delay disease progression and increase survival. It is clear that newer combinations – particularly three drug combinations such as RVd (Revlimid-Velcade-dex) and VMP (Velcade-melphalan-prednisone) are associated with 80-100% response rates, and up to 40% CR rates when used front-line, which is an exponential increase over older combinations such as MP and VAD, but these trials are so young that for many, median survival and time to disease progression, never mind overall survival, have not yet been reached.
Until we have these answers it is important that patients, particularly those who are newly diagnosed and untreated, consider one of the many clinical trials available (www.myelomatrials.org) so that we can answer these critical questions.In the meantime, it is also important to understand that three and four drug combinations are likely to confer the greatest benefit to patients, the trials have very short follow-up, and many have limited patients analyzed to date, so it is premature to make any conclusions about what the optimal combination, at any point, may be.
May 19, 2009
Translational Research – Under Construction! Louise M. Perkins, Ph.D., Chief Scientific Officer
How should the next generation of medicines be discovered and advanced to clinical testing? Who should pay for and conduct this work? These were among the key topics at the Translational Medicine Alliance Forum that I attended in Philadelphia on May 14 and 15. This forum brought together thought leaders from foundations, academia, government and the private sector to identify barriers, possible solutions and the role that each of these key stakeholders should play in this important field.
So, what is translational research and why is it so important? It is the process of advancing new medical approaches from the research laboratory to patients– that is, translating research “from bench to bedside”. The MMRF has long supported this type of research, and the MMRF Scientific Agenda charts strategies aimed at translating an understanding of the genomics and proteomics of myeloma to targets and then to treatments and personalized medicine. This may sound easy on paper - but actually making it happen in an efficient way is the real challenge.
What was clear from this meeting is that disease-focused foundations like the MMRF are viewed as critical players in this complex, expensive and time-consuming process. I was privileged to be invited as a panelist discussing the role of non-profit foundations in translational research along with other venture philanthropy leaders, like Bob Beall - MMRF Board Member and head of the Cystic Fibrosis Foundation (http://www.translationalmedicinealliance.org/forum/agenda.cfm).
While the specific approaches of the different groups vary, the common thread is that leading non-profit foundations like the MMRF fund commercial as well as academic research to overcome the barriers related to bringing new treatments forward. The MMRF and its sister organization the MMRC have already made tremendous advances, such as opening clinical trials 30%-40% more quickly than the industry standard. But, despite these improvements, there are still significant needs. For example, there is a growing urgency to devise and fund new solutions that incentivize clinical development of drugs in MM since financial constraints facing the entire pharma and biotech industry threaten continued innovation and progress.
Commercial investors who provide funding for the development of innovative products were also at the meeting. These venture capitalists uniformly acknowledged that programs like the MMRF Biotech Investment Award that support promising biotech drugs are not just a source of cash. Importantly, they also represent a “seal of approval” that can trigger the influx of venture capital to those companies. Why is this important? Simply put, it means that MMRF donor dollars are complemented with additional money that might not otherwise come to the field of myeloma thereby extending our reach and enhancing the chance of success for those drugs.
All in all, these were an exciting two days at the Translational Medicine Alliance Forum and it was rewarding to see so many scientists and business people working diligently to develop increasingly optimized models to translate research findings to real-world therapies for all patients, including those with multiple myeloma.
